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Orphan Drugs: Research and Reviews, Volume 5, Issue (2015)

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Orphan Drugs: Research and Reviews, Volume 5, Issue (2015)

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Review

Review Article

Orphan drugs in development for Huntington's disease: challenges and progress

Jean-Marc Burgunder

Pages: 1-9

Published online: 12 Feb 2015

AbstractforOrphan drugs in development for Huntington's disease: challenges and progress | Full Text | References | PDF (228.1 KB)

70 Views
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Open Access

Review Article

Update on the challenges of treating trigeminal neuralgia

Mark Obermann

Pages: 11-17

Published online: 10 Apr 2015

AbstractforUpdate on the challenges of treating trigeminal neuralgia | Full Text | References | PDF (177.7 KB)

81 Views
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Open Access

Review Article

Amyotrophic lateral sclerosis: clinical perspectives

Verena C Haringer & Summer B Gibson

Pages: 19-31

Published online: 15 Apr 2015

AbstractforAmyotrophic lateral sclerosis: clinical perspectives | Full Text | References | PDF (308.1 KB)

63 Views
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Open Access

Review Article

Orphan drugs in development for long-chain fatty acid oxidation disorders: challenges and progress

Angela Sun & J Lawrence Merritt II

Pages: 33-41

Published online: 28 Apr 2015

AbstractforOrphan drugs in development for long-chain fatty acid oxidation disorders: challenges and progress | Full Text | References | PDF (375.2 KB)

73 Views
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Open Access

Review Article

Fahr's disease: current perspectives

Xin You Tai & Amit Batla

Pages: 43-49

Published online: 16 Jul 2015

AbstractforFahr's disease: current perspectives | Full Text | References | PDF (578.9 KB)

252 Views
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Open Access

Review Article

Drugs in development and dietary approach for Duchenne muscular dystrophy

Corrado Angelini & Elisabetta Tasca

Pages: 51-60

Published online: 11 Aug 2015

AbstractforDrugs in development and dietary approach for Duchenne muscular dystrophy | Full Text | References | PDF (1.1 MB)

75 Views
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Open Access

Review Article

Profile of lumacaftor/ivacaftor combination: potential in the treatment of cystic fibrosis

Alexandria M Arends & Rebecca S Pettit

Pages: 61-68

Published online: 14 Aug 2015

AbstractforProfile of lumacaftor/ivacaftor combination: potential in the treatment of cystic fibrosis | Full Text | References | PDF (404.8 KB)

73 Views
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Open Access

Original Research

Article

The efficacy and safety of adalimumab in ocular inflammatory disease

Cheryl A Arcinue, Khayyam Durrani, Pichaporn Artornsombudh, Alaa Radwan, Ravi Parikh, Ana M Suelves, Sana S Siddique, Ian Chang, Janine M Preble & Charles Stephen Foster

Pages: 69-74

Published online: 31 Aug 2015

AbstractforThe efficacy and safety of adalimumab in ocular inflammatory disease | Full Text | References | PDF (252.4 KB)

62 Views
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Open Access

Review

Review Article

Paroxysmal nocturnal hemoglobinuria: new concepts in pathophysiology and treatment

Ilene Ceil Weitz

Pages: 75-81

Published online: 01 Sep 2015

AbstractforParoxysmal nocturnal hemoglobinuria: new concepts in pathophysiology and treatment | Full Text | References | PDF (193.7 KB)

64 Views
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Open Access

Review Article

Orphan drugs in development for primary biliary cirrhosis: challenges and progress

Ahmad H Ali, Thomas J Byrne & Keith D Lindor

Pages: 83-97

Published online: 10 Sep 2015

AbstractforOrphan drugs in development for primary biliary cirrhosis: challenges and progress | Full Text | References | PDF (305.7 KB)

77 Views
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Open Access

Review Article

Orphan drugs for sickle vaso-occlusion: dawn of a new era of targeted treatment

Carlton Dampier

Pages: 99-112

Published online: 02 Nov 2015

AbstractforOrphan drugs for sickle vaso-occlusion: dawn of a new era of targeted treatment | Full Text | References | PDF (629.3 KB)

76 Views
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Open Access

Review Article

Denosumab in the treatment of hypercalcemia secondary to malignancy

Alan C Dalkin & Mitchell H Rosner

Pages: 113-121

Published online: 23 Nov 2015

AbstractforDenosumab in the treatment of hypercalcemia secondary to malignancy | Full Text | References | PDF (809.9 KB)

82 Views
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Open Access

Explore articles

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Orphan drugs for the treatment of aspergillosis: focus on isavuconazole

Samantha E Jacobs et al.

Review Article | Published online: 3 May 2017

Open Access

Nitisinone: a review

A Cigdem Aktuglu-Zeybek et al.

Review Article | Published online: 31 Jan 2017

Open Access

Spotlight on anagrelide hydrochloride for the treatment of essential thrombocythemia

Anita Sarma et al.

Review Article | Published online: 31 Jan 2017

Open Access

Pompe disease: clinical perspectives

Juan Francisco Cabello et al.

Review Article | Published online: 28 Dec 2016

Open Access

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Orphan Drugs: Research and Reviews (Vol.5, Iss., 2015)

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